Nanoparticle-mediated gene transfer specific to retinal pigment epithelial cells

Safety and Efficacy of Gene Transfer for Leber's Congenital Amaurosis

Albert M. Maguire, Francesca Simonelli, Eric A. Pierce et al. · New England Journal of Medicine · 2008 · 2.1K citations · Full text

Ocular Disease, Genetics, Immunology +18

Effect of Gene Therapy on Visual Function in Leber's Congenital Amaurosis

James Bainbridge, Alexander J. Smith, Susie S. Barker et al. · New England Journal of Medicine · 2008 · 1.9K citations · Full text

Gene Therapies, Ocular Disease, Retina +14

Treatment of Leber Congenital Amaurosis Due to <i>RPE65</i> Mutations by Ocular Subretinal Injection of Adeno-Associated Virus Gene Vector: Short-Term Results of a Phase I Trial

William W. Hauswirth, Tomás S. Alemán, Shalesh Kaushal et al. · Human Gene Therapy · 2008 · 937 citations · Full text

Effect of Genome Size on AAV Vector Packaging

Zhijian Wu, Hongyan Yang, Peter Colosi · Molecular Therapy · 2009 · 935 citations · Full text

Viral Replication, Aav Vector Genomes, Vector Genomes +17

Age-dependent effects of RPE65 gene therapy for Leber's congenital amaurosis: a phase 1 dose-escalation trial

Albert M. Maguire, Katherine A. High, Alberto Auricchio et al. · The Lancet · 2009 · 849 citations

Gene Therapies, Genetic Disorder, Genetics +7